Gene Technology: Gene Therapy
Assignment 2: Gene Technology
Due Week 8 and worth 120 points
Gene technology carries with it social and ethical implications—many of which engender personal views and discussion.
Select one (1) of the following biotechnology topics to write about:
Genetically modified crop plants
Genetically modified microorganisms
Genetically modified animals
Personal genomics and / or personalized medicine for humans
Gene therapy
Write a four to six (4 to 6) page paper on your chosen topic. Organize your paper into sections corresponding to the following requirements:
Biological basis. Describe the technology. Discuss what it accomplishes. Elaborate on the scientific principles that make this technology possible. Your goal in this section of the paper is to show the instructor that you understand the underlying science behind the technology. Describe how exactly the technology works. Discuss the biological principles that underlie this technology.
Social and ethical implications. Without disclosing your personal view about this technology, provide an analysis of its social and ethical implications. State the ethical concerns apparent in the use of this technology. Discuss the benefits and risks. Your goal in this section is to look at all sides of the issue. In the next section, you will give your opinion.
Personal viewpoint. In the previous section, your goal was to be as objective as possible, to look at all sides of the issues. In this section, your goals are to give a personal opinion about the technology and provide a justification of that opinion.
Use at least three (3) quality resources in this assignment, in addition to the course text. Note: Wikipedia and similar Websites do not qualify as quality resources. The body of the paper must have in-text citations that correspond to the references. Integrate all sources into your paper using proper techniques of quoting, paraphrasing and summarizing, along with proper use of in-text citations to credit your sources.
Your assignment must follow these formatting requirements:
Be typed, double spaced, using Times New Roman font (size 12), with one-inch margins on all sides; citations and references must follow APA or school-specific format. Check with your professor for any additional instructions.
Include a cover page containing the title of the assignment, the student's name, the professor's name, the course title, and the date. The cover page and the reference page are not included in the required assignment page length.
Name:
University:
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Gene Technology: Gene Therapy
1 Biological Basis of Gene Therapy
1 Description of the technology and how it works
The purpose of gene therapy is to introduce a gene into cells to either replace the defective genes or synthesize the intended functional protein (Mammen, 2007). It is a technique used to introduce a normal copy of a gene for protein function restoration, particularly if a mutation of the defective gene results in faulty or missing protein. To perform gene therapy, scientists insert genes directly into cells which have lost their functions. Genetically engineered vectors such as non-pathogenic viruses act as carriers to deliver the normal genes into cells by infecting them (U.S. National Library of Medicine, 2014). Viruses, such as retroviruses, are usually engineered to make them non-virulent in human cells. The viruses integrate their genetic material and the new gene to be transferred with the human chromosome in the target cell. However, in adenoviruses, the viral DNA is introduced into the cell nucleus without integrating the DNA into the human chromosome.
1.2 How the technology works
Vectors carrying the desired gene can be administered intravenously into the target tissues and taken up by specific cells. Alternatively, cells can be extracted from the patient and then exposed to the vectors in the lab (U.S. National Library of Medicine, 2014). The cells that have been infected by the vectors are then injected into the cells of the patient. Effective treatment of the patient will be demonstrated when the new gene successfully delivered by the vector will synthesize a functional protein as shown in Fig 1. During the process of gene therapy, scientists encounter several hurdles which they must overcome if the technique is to be successful in the treatment of diseases. For instance, better ways of gene delivery and specificity to target cells has to be sought if this process is to be effective. In addition, the new genes introduced into cells have to be controlled precisely in the human body/patient.
Fig 1. Gene therapy using adenovirus (U.S. National Library of Medicine, 2014).
1 What gene therapy accomplishes
Still in its experimental phase, the technology uses genes to either prevent or treat diseases. It is hoped that doctors will be able to treat diseases by inserting genes into the cells of patients instead of using drugs or performing any surgical operation. The technique accomplishes the intended purpose by introducing a novel gene which helps the body to fight an infection, knocking out or inactivating the mutated gene that expresses a nonfunctioning protein, or by replacing a mutated gene which causes the infection with a healthy one (Mammen et al, 2007). Gene therapy has the potential to treat a variety of diseases such as cancers, viral infections, and genetic diseases. However, since the technique is considered to be risky, the effectiveness and safety of gene therapy can only be tested for the treatment of infections that currently lack cures.
2 Scientific principles making the gene therapy possible
The genes, which are contained in chromosomes, are the basic unit of heredity. These units are arranged in base sequences which provide instructions for making proteins. Genetic diseases often result from an alteration in these basic units of heredity. In gene therapy, new genetic materials are introduced into the cells to either help cells synthesize functional proteins or compensate for the defective genes (Mammen et al, 2007).
2 Social and ethical implications of gene therapy
2 Ethical concerns in the adoption of gene therapy
There are a number of important questions that need to be addressed before adopting the technology of gene therapy in the society. The question about what is accepted as normal in the society and what defines disabilities or disorders remains unanswered (Mammen et al, 2007). It is also question about if disabilities are diseases, and if they need to be prevented or cured. Since gene therapy targets genetic disorders, the efforts to search for cures would be see to demean the lives of those living with disabilities. There are two approaches to gene therapy: somatic and germ line gene therapy. Somatic gene therapy targets the adult cells of the patient while germ line gene therapy targets the sex cells to prevent the defective gene to be passes to other generations. It is not yet established whether somatic gene therapy is more or less ethical than germ line type. The issue of cost has also been debated (Mammen et al, 2007). The technology is expensive, and only a few individuals will have access to treatments.
3 Benefits and risks
The benefits of gene therapy is that it is able to restore someone born with the disease to a disease free state. Individuals with genetic diseases are afforded another chance to live normal lives (Nienhuis, 2008). While the technique has been praised for this role, the risks to adopting gene therapy have derailed its wide usage in the modern society. The technology poses serious potential risks to human health and these challenges need to be addressed. Gene delivery, durability and integration, immune responses, and safety of the vectors are some of the common challenges preventing faster adoption of gene therapy.
While the technique has been used to treat patients with genetic disorders such as X-linked severe combined immunodeficiency (X-SCID), it is not yet predictable if genes will be delivered successfully, even in patients with single-gene diseases (Mammen et al, 2007). In some disorders, durability and integration pose serious challenges particularly when the genetic material is required to remain functional for some expected duration. Another risk common in gene therapy is the immune responses elicited by the host immune if viral vectors are used in gene delivery. These vectors may be recognized by the immune cells as foreign and hence attack them. While such responses are targets for cancer treatment, the attacks may reduce the intended efficacy of gene therapy. Another important risk of gene therapy concerns the safety of the vectors used in delivering gees. Immune and inflammatory responses, toxicity and gene targeting and control issues have been observed as common problems in gene therapy. There have also been fears that such viruses may regain their virulent nature in the human cells (Mammen et al, 2007).
3 Personal viewpoint and justification
Gene therapy is a revolutionary technology capable of restoring individuals born with serious genetic diseases to normal life. The technique has a potential to cure serious viral diseases, cancer...